Induced pluripotent stem cells for regenerative cardiovascular therapies and biomedical discovery

Advanced Drug Delivery Reviews

Volumn 63, Issue 4, 2011, Pages 324-330

  Nsair, Ali ^a   MacLellan, W Robb ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Biomedical discovery; Embryonic stem cells; Induced pluripotent stem cells; Regenerative therapies; Stem cells

Indexed keywords

BIOMEDICAL DISCOVERY; DRUG DISCOVERY; DRUG SCREENING; EMBRYONIC STEM CELLS; ETHICAL CONCERNS; HIGH THROUGHPUT; IN-VITRO; PLURIPOTENT; PLURIPOTENT STEM CELLS; REGENERATIVE THERAPIES; SOMATIC CELLS; STEM CELL BIOLOGY;

DRUG THERAPY; STEM CELLS;

CYTOLOGY;

ANGIOGENESIS; CARDIOTOXICITY; CARDIOVASCULAR DISEASE; CELL DIFFERENTIATION; CELL NUCLEUS TRANSPLANTATION; CELL RENEWAL; DISEASE MODEL; DRUG RESEARCH; DRUG SAFETY; DRUG SCREENING; EMBRYONIC STEM CELL; GENE EXPRESSION PROFILING; HAPLOTYPE; HEART INFARCTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOPHILIA A; HIGH THROUGHPUT SCREENING; HUMAN; MEDICAL ETHICS; MULTIPOTENT STEM CELL; NONHUMAN; NUCLEAR REPROGRAMMING; ORGAN INJURY; PARKINSON DISEASE; PATHOPHYSIOLOGY; PATIENT SAFETY; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; QT PROLONGATION; REGENERATIVE MEDICINE; REVIEW; SICKLE CELL ANEMIA; SOMATIC CELL; STEM CELL TRANSPLANTATION;

ANIMALS; CARDIOVASCULAR DISEASES; CELL DIFFERENTIATION; DRUG DISCOVERY; HIGH-THROUGHPUT SCREENING ASSAYS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; NUCLEAR REPROGRAMMING; REGENERATIVE MEDICINE;

EID: 79957788311     PISSN: 0169409X     EISSN: 0169409X     Source Type: Journal    
DOI: 10.1016/j.addr.2011.01.013     Document Type: Review

References (63)

1. Becker A.J., Till J.E. Cytological demonstration of the clonal nature of spleen colonies derived from transplanted mouse marrow cells. Nature 1963, 197:452-454.
2. Evans M.J. Establishment in culture of pluripotential cells from mouse embryos. Proc. Natl Acad. Sci. USA 1981, 78(12):7634-7638.
3. King T.J. Transplantation of living nuclei from blastula cells into enucleated frogs' eggs. Proc. Natl Acad. Sci. USA 1952, 38:455-463.
4. Schnieke A.E., Ritchie W.A., Mycock K., Scott A.R., Ritchie M., Wilmut I., Colman A., Campbell K.H. Human factor IX transgenic sheep produced by transfer of nuclei from transfected fetal fibroblasts. Science 1997, 278(5346):2130-2133.
5. Gurdon J.B., Simonsson S. Nuclear reprogramming and stem cell creation. Proc. Natl Acad. Sci. USA 2003, 100(1):11819-11822.
6. Byrne J.A., Clepper L.L., Nelson M., Sanger W.G., Gokhale S., Wolf D.P., Mitalipov S.M. Producing primate embryonic stem cells by somatic cell nuclear transfer. Nature 2007, 450:497-502.
7. Martin G. Isolation of a pluripotent cell line from early mouse embryos cultured in medium conditioned by teratocarcinoma stem cells. Proc. Natl Acad. Sci. USA 1981, 78(12):7634-7638.
8. Thomson J., Shapiro S., Waknitz M., Swiergiel J., Marshall V., Jones J. Embryonic stem cell lines derived from human blastocysts. Science 1998, 282(5391):1145-1147.
9. World's first clinical trial of human embryonic stem cell therapy cleared. Regen. Med. 2009, 4(2):161. Geron Corporation.
10. Takahashi K. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006, 126(4):663-676.
11. Yu J., Smuga-Otto K., Antosiewicz-Bourget J., Frane J.L., Tian S., Nie J., Jonsdottir G.A., Ruotti V., Stewart R., Slukvin I.I., Thomson J.A. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007, 318:1917-1920.
12. Hochedlinger K. Epigenetic reprogramming and induced pluripotency. Development 2009, 136(4):509-523.
13. Woltjen K., Mohseni P., Desai R., Mileikovsky M., Hämäläinen R., Cowling R., Wang W., Liu P., Gertsenstein M., Kaji K., Sung H.K., Nagy A. piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature 2009, 489(7239):766-770.
14. Zhou H., Zhu S., Joo J.Y., Do J.T., Xiong W., Kim J.B., Zhang K., Schöler H.R., Ding S. Conversion of mouse epiblast stem cells to an earlier pluripotency state by small molecules. J. Biol. Chem. 2010, 285(39):29676-29680.
15. Warren L., Ahfeldt T., Loh Y.H., Li H., Lau F., Ebina W., Mandal P.K., Smith Z.D., Meissner A., Daley G.Q., Brack A.S., Collins J.J., Cowan C., Schlaeger T.M., Rossi D.J. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 2010, 7(5):618-630.
16. Okita K., Ichisaka T., Yamanaka S. Generation of germline-competent induced pluripotent stem cells. Nature 2007, 448:313-317.
17. Takahashi K., Ohnuki M., Narita M., Ichisaka T., Tomoda K., Yamanaka S. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007, 131:861-872.
18. Boland Michael J., Nazor Kristopher L., Rodriguez Alberto R., Gifford Wesley, Martin Greg, Kupriyanov Sergey, Baldwin Kristin K. Adult mice generated from induced pluripotent stem cells. Nature 2009, 461:91-94.
19. Deng J., Xie B., Gore A., LeProust E.M., Antosiewicz-Bourget J., Egli D., Maherali N., Park I.H., Yu J., Daley G.Q., Eggan K., Hochedlinger K., Thomson J., Wang W., Gao Y., Zhang K. Targeted bisulfite sequencing reveals changes in DNA methylation associated with nuclear reprogramming. Nat. Biotechnol. 2009, 27:353-360.
20. Chin M.H., Xie W., Volinia S., Singer M., Peterson C., Ambartsumyan G., Aimiuwu O., Richter L., Zhang J., Khvorostov I., Ott V., Grunstein M., Lavon N., Benvenisty N., Croce C.M., Clark A.T., Baxter T., Pyle A.D., Teitell M.A., Pelegrini M., Plath K., Lowry W.E. Induced pluripotent stem cells and embryonic stem cells are distinguished by gene expression signatures. Cell Stem Cell 2009, 5:111-123.
21. Kim K., Wen B., Ng K., Zhao R., Cahan P., Kim J., Aryee M.J., Ji H., Ehrlich L.I., Yabuuchi A., Takeuchi A., Cunniff K.C., Hongguang H., McKinney-Freeman S., Naveiras O., Yoon T.J., Irizarry R.A., Jung N., Seita J., Hanna J., Murakami P., Jaenisch R., Weissleder R., Orkin S.H., Weissman I.L., Feinberg A.P., Daley G.Q. Epigenetic memory in induced pluripotent stem cells. Nature 2010, 467(7313):285-290.
22. Ghosh Z., Wu Y., Hu S., Quertermous T., Wu J.C. Persistent donor cell gene expression among human induced pluripotent stem cells contributes to differences with human embryonic stem cells. PLoS ONE 2010, 5(2):e8975.
23. Miura K., Aoi T., Okada A., Takahashi K., Okita K., Nakagawa M., Koyanagi M., Tanabe K., Ohnuki M., Ogawa D., Ikeda E., Okano H., Yamanaka S. Variation in the safety of induced pluripotent stem cell lines. Nat. Biotechnol. 2009, 27:743-745.
24. Park I.H., West J.A., Yabuuchi A., Huo H., Ince T.A., Lerou P.H., Lensch M.W., Daley G.Q. Reprogramming of human somatic cells to pluripotency with defined factors. Nature 2008, 451:141-146.
25. Ciceri F., Stanghellini M.T., Bondanza A., Traversari C., Salomoni M., Turchetto L., Colombi S., Bernardi M., Peccatori J., Pescarollo A., Servida P., Magnani Z., Perna S.K., Valtolina V., Crippa F., Callegaro L., Spoldi E., Crocchiolo R., Fleischhauer K., Ponzoni M., Vago L., Rossini S., Santoro A., Todisco E., Apperley J., Olavarria E., Slavin S., Weissinger E.M., Ganser A., Stadler M., Yannaki E., Fassas A., Anagnostopoulos A., Bregni M., Stampino C.G., Bruzzi P., Bordignon C. Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study. Lancet Oncol. 2009, 10(5):489-500.
26. Menzel O., Wildhaber B.E., Jond C., Lasne F., Habre W., Trono D., Nguyen T.H., Chardot C. Biosafety in ex vivo gene therapy and conditional ablation of lentivirally transduced hepatocytes in nonhuman primates. Mol. Ther. 2009, 17(10):1754-1760.
27. McCormick J.B. Stem cells and ethics: current issues. J. Cardiovasc. Transl. Res. 2010, 3(2):122-127.
28. Meyer J. The significance of induced pluripotent stem cells for basic research and clinical therapy. J. Med. Ethics 2008, 34(12):849-851.
29. Hanna J., Markoulaki S., Sun C.W., Meissner A., Cassady J.P., Beard C., Brambrink T., Wu L.C., Townes T.M., Jaenisch R. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 2007, 318(5858):1920-1923.
30. Wernig M., Pruszak J., Hedlund E., Fu D., Soldner F., Broccoli V., Constantine-Paton M., Isacson O., Jaenisch R. Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease. Proc. Natl Acad. Sci. USA 2008, 105:5656-5861.
31. Xu D., Fink L.M., Adcock D.M., Yang J., Ward D.C., Ma Y. Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc. Natl Acad. Sci. USA 2009, 106:808-813.
32. Nelson T.J., Yamada S., Perez-Terzic C., Ikeda Y., Terzic A. Repair of acute myocardial infarction by human stemness factors induced pluripotent stem cells. Circulation 2009, 120:408-416.
33. Roberts R.M., Ezashi T. Induced pluripotent stem cells from swine (Sus scrofa): why they may prove to be important. Cell Cycle 2009, 8(19):3078-3081.
34. Hutcheson K.A., Hueman M.T., Hopkins M.B., Glower D.D., Taylor D.A. Comparison of benefits on myocardial performance of cellular cardiomyoplasty with skeletal myoblasts and fibroblasts. Cell Transplant. 2009, 9:359-368.
35. Galli D., Staszewsky L., Zanetta L., Sampaolesi M., Bai A., Martinoli E., Carlo E., Balconi G., Fiordaliso F., Chimenti S., Cusella G., Dejana E., Cossu G., Latini R. Mesoangioblasts, vessel-associated multipotent stem cells, repair the infracted heart by multiple cellular mechanisms: a comparison with bone marrow progenitors, fibroblasts, and endothelial cells. Arterioscler. Thromb. Vasc. Biol. 2005, 25:692-697.
36. Laflamme M.A., Epstein S.E., Murry C.E. Cell-based therapy for myocardial ischemia and infarction: pathophysiological mechanisms. Annu. Rev. Pathol. 2007, 2:307-339.
37. Nishikawa S.I., Hirashima M., Matsuyoshi N., Kodama H. Progressive lineage analysis by cell sorting and culture identifies FLK1+VE-cadherin+ cells at a diverging point of endothelial and hemopoietic lineages. Development 1998, 125:1747-1757.
38. Menasché P. Stem cell therapy for heart failure. Are arrhythmias a real safety concern?. Circulation 2009, 119:2735-2740.
39. Min J.Y., Sullivan M.F., Ke Q., Converso K.L., Chen Y., Morgan J.P., Xiao Y.F. Long-term improvement of cardiac function in rats after infarction by transplantation of embryonic stem cells. J. Thorac. Cardiovasc. Surg. 2003, 125:361-369.
40. Singla D.K., Ma L., Douglas P.S., Sullivan R., Lyons G.E., Kamp T.J. Transplantation of embryonic stem cells into the infarcted mouse heart: formation of multiple cell types. J. Mol. Cell. Cardiol. 2006, 40:195-200.
41. Nussbaum J., Laflamme M.A., Virag J.A., Ware C.B., Masino A., Muskheli V., Pabon L., Reinecke H., Murry C.E. Transplantation of undifferentiated murine embryonic stem cells in the heart: teratoma formation and immune response. FASEB J. 2007, 21:1345-1357.
42. Behfar A., Faustino R.S., Arrell D.K., Hodgson D.M., Yamada S., Puceat M., Niederlander N., Alekseev A.E., Zingman L.V., Terzic A. Cardiopoietic programming of embryonic stem cells for tumor-free heart repair. J. Exp. Med. 2007, 204:405-420.
43. Caspi O., Kehat I., Habib M., Arbel G., Gepstein A., Yankelson L., Aronson D., Beyar R., Gepstein L. Transplantation of human embryonic stem cell-derived cardiomyocytes improves myocardial performance in infarcted rat hearts. J. Am. Coll. Cardiol. 2007, 50:1884-1893.
44. Swijnenburg R.J., Vogel H., Baker J., Kofidis T., Gunawan F., Lebl D.R., Caffarelli A.D., de Bruin J.L., Fedoseyeva E.V., Robbins R.C. Embryonic stem cell immunogenicity increases upon differentiation after transplantation into ischemic myocardium. Circulation 2005, 112:166-172.
45. Schenke-Layland K., Angelis E., Butylkova Y., Heydarkhan-Hagvall S., Gekas C., Zhang R., Goldhaber J.I., Mikkola H.K., Plath K., MacLellan W.R. Reprogrammed mouse fibroblasts differentiate into cells of the cardiovascular and hematopoietic lineages. Stem Cells 2008, 26(6):1537-1546.
46. Narazaki G., Teranishi M., Okita K., Kim B., Matsuoka S., Yamanaka S., Yamashita J.K. Directed and systematic differentiation of cardiovascular cells from mouse induced pluripotent stem cells. Circulation 2008, 118(5):498-506.
47. Zwi L., Arbel G., Huber I., Gepstein A., Park I.H., Gepstein L. Cardiomyocyte differentiation of human induced pluripotent stem cells. Circulation 2009, 120(15):1513-1523.
48. Zhang J., Soerens A.G., Koonce C.H., Yu J., Palecek S.P., Thomson J.A., Kamp T.J. Functional cardiomyocytes derived from human induced pluripotent stem cells. Circ. Res. 2009, 104(4):e30-e41.
49. Martinez-Fernandez A., Yamada S., Reyes S., Alekseev A.E., Perez-Terzic C., Ikeda Y., Terzic A. iPS programmed without c-MYC yield proficient cardiogenesis for functional heart chimerism. Circ. Res. 2009, 105(7):648-656.
50. Nelson Timothy J., Almudena Martinez-Fernandez, Satsuki Yamada, Carmen Perez-Terzic, Yasuhiro Ikeda, Andre Terzic Repair of acute myocardial infarction by human stemness factors induced pluripotent stem cells. Circulation 2009, 120:408-416.
51. Osafune K., Borowiak M., Martinez R.J., Fitz-Gerald C.S., Sato Y., Cowan C.A., Chien K.R., Melton D.A. Marked differences in differentiation propensity among human embryonic stem cell lines. Nat. Biotechnol. 2008, 26(3):313-315.
52. Pickering S.J., Patel M., Taylor H., Black C., Burns C.J., Ekonomou A., Braude P.R. Generation of a human embryonic stem cell line encoding the cystic fibrosis mutation deltaF508, using preimplantation genetic diagnosis. Reprod. Biomed. Online 2005, 10(3):390-397.
53. Stephenson E.L., Braude P.R. Preimplantation genetic diagnosis as a source of human embryonic stem cells for disease research and drug discovery. BJOG 2009, 116(2):158-165.
54. Moretti A., Nakano A., Lam J.T., Bernshausen A., Chen Y., Qyang Y., Bu L., Sasaki M., Martin-Puig S., Sun Y., Evans S.M., Laugwitz K.L., Chien K.R. Multipotent embryonic isl1+ progenitor cells lead to cardiac, smooth muscle, and endothelial cell diversification. Cell 2006, 127(6):1151-1165.
55. Dimos J.T., Niakan K.K., Weisenthal L.M., Mitsumoto H., Chung W., Croft G.F., Saphier G., Leibel R., Goland R., Wichterle H., Henderson C.E., Eggan K. Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science 2008, 321(5893):1169-1170.
56. Ebert A.D., Rose F.F., Mattis V.B., Lorson C.L., Thomson J.A., Svendsen C.N. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 2009, 457(7227):227-280.
57. Ye Zhaohui, Mali Prashant, Dowey Sarah, Williams Donna M., Jang Yoon-Young, Dang Chi V., Spivak Jerry L., Moliterno Alison R., Cheng Linzhao Human induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders. Blood 2009, 114(27):5473-5480.
58. Hughes B. 2008 FDA drug approvals. Nat. Rev. Drug Discov. 2009, 8(93).
59. Hu Michael, Sheu Jack, Tschopp Daniel The Innovation Gap in Pharmaceutical Drug Discovery and New Models for R&D Success 2007, Kellogg School of Management.
60. Kola I. Can the pharmaceutical industry reduce attrition rates?. Nat. Rev. Drug Discov. 2004, 3:711-716.
61. Pugsley M.K., Curtis M.J. Principles of safety pharmacology. Br. J. Pharmacol. 2008, 154(7):1382-1399.
62. Braam S.R. Human stem cell models for predictive cardiac safety pharmacology. Stem Cell Res. 2010, 4(3):155-156.
63. Otsuji T.G., Kurose Y., Yamauchi K., Tada M., Nakatsuji N. Progressive maturation in contracting cardiomyocytes derived from human embryonic stem cells: qualitative effects on electrophysiological responses to drugs. Stem Cell Res. 2010, 4(3):201-213.